Enhancing insights into pulmonary vascular disease through a precision medicine approach: A joint nhlbi-cardiovascular medical research and education fund workshop report

John H. Newman, Stuart Rich, Steven H. Abman, John H. Alexander, John Barnard, Gerald J. Beck, Raymond L. Benza, Todd M. Bull, Stephen Y. Chan, Hyung J. Chun, Declan Doogan, Jocelyn Dupuis, Serpil C. Erzurum, Robert P. Frantz, Mark Geraci, Hunter Gillies, Mark Gladwin, Michael P. Gray, Anna R. Hemnes, Roy S. HerbstAdrian F. Hernandez, Nicholas S. Hill, Evelyn M. Horn, Kendall Hunter, Zhi Cheng Jing, Roger Johns, Sanjay Kaul, Steven M. Kawut, Tim Lahm, Jane A. Leopold, Greg D. Lewis, Stephen C. Mathai, Vallerie V. McLaughlin, Evangelos D. Michelakis, Steven D. Nathan, William Nichols, Grier Page, Marlene Rabinovitch, Jonathan Rich, Franz Rischard, Sharon Rounds, Sanjiv J. Shah, Victor F. Tapson, Naomi Lowy, Norman Stockbridge, Gail Weinmann, Lei Xiao

Research output: Contribution to journalArticle

Abstract

The Division of Lung Diseases of the NHLBI and the Cardiovascular Medical Education and Research Fund held a workshop to discuss how to leverage the anticipated scientific output from the recently launched "Redefining Pulmonary Hypertension through Pulmonary Vascular Disease Phenomics" (PVDOMICS) program to develop newer approaches to pulmonary vascular disease. PVDOMICS is a collaborative, protocol-driven network to analyze all patient populations with pulmonary hypertension to define novel pulmonary vascular disease (PVD) phenotypes. Stakeholders, including basic, translational, and clinical investigators; clinicians; patient advocacy organizations; regulatory agencies; and pharmaceutical industry experts, joined to discuss the application of precision medicine to PVD clinical trials. Recommendations were generated for discussion of research priorities in line with NHLBI Strategic Vision Goals that include: (1) A national effort, involving all the stakeholders, should seek to coordinate biosamples and biodata from all funded programs to a web-based repository so that information can be shared and correlated with other research projects. Example programs sponsored by NHLBI include PVDOMICS, Pulmonary Hypertension Breakthrough Initiative, the National Biological Sample and Data Repository for PAH, and the National Precision Medicine Initiative. (2) A task force to develop a master clinical trials protocol for PVD to apply precision medicine principles to future clinical trials. Specific features include: (a) adoption of smaller clinical trials that incorporate biomarker-guided enrichment strategies, using adaptive and innovative statistical designs; and (b) development of newer endpoints that reflect well-defined and clinically meaningful changes. (3) Development of updated and systematic variables in imaging, hemodynamic, cellular, genomic, and metabolic tests that will help precisely identify individual and shared features of PVD and serve as the basis of novel phenotypes for therapeutic interventions.

Original languageEnglish (US)
Pages (from-to)1661-1670
Number of pages10
JournalAmerican journal of respiratory and critical care medicine
Volume195
Issue number12
DOIs
StatePublished - Jun 15 2017

Keywords

  • Master protocol
  • Precision medicine
  • Pulmonary hypertension
  • Pulmonary vascular disease
  • Pulmonary vascular disease phenomics

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Critical Care and Intensive Care Medicine

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    Newman, J. H., Rich, S., Abman, S. H., Alexander, J. H., Barnard, J., Beck, G. J., Benza, R. L., Bull, T. M., Chan, S. Y., Chun, H. J., Doogan, D., Dupuis, J., Erzurum, S. C., Frantz, R. P., Geraci, M., Gillies, H., Gladwin, M., Gray, M. P., Hemnes, A. R., ... Xiao, L. (2017). Enhancing insights into pulmonary vascular disease through a precision medicine approach: A joint nhlbi-cardiovascular medical research and education fund workshop report. American journal of respiratory and critical care medicine, 195(12), 1661-1670. https://doi.org/10.1164/rccm.201701-0150WS